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Ultragenyx Announces Phase 1 Results of UX001 in Hereditary Inclusion Body Myopathy (HIBM), a Rare Neuromuscular Disease
Date:5/1/2012

ng from HIBM," said Emil D. Kakkis, MD, PhD, Chief Executive Officer of Ultragenyx. "Based on these favorable Phase 1 results, we look forward to moving quickly to initiate a Phase 2 study of UX001." 

Ultragenyx plans to present the Phase 1 data at the 17th Annual International World Muscle Society Congress in Perth, Australia later this year.

About HIBM  

HIBM is also known as GNE myopathy, distal myopathy with rimmed vacuoles (DMRV) and Nonaka disease.  HIBM is a severe, adult-onset, progressive, genetic neuromuscular disease caused by a deficiency of an enzyme in the first step of sialic acid biosynthesis needed for the modification of proteins and fats. Patients with HIBM typically begin to have weakness and abnormal walking at 18 to 30 years of age. Over the ensuing 10 to 20 years, many patients progressively lose significant functional ability and become wheelchair-bound. There are no current treatments for this disease.

About Ultragenyx

Ultragenyx is a privately held, developmental stage biotechnology company committed to bringing life-enhancing therapeutics for patients with rare and ultra-rare genetic diseases, also known as orphan and ultra-orphan diseases, to market.  The company focuses on rare metabolic diseases that affect small numbers of patients, but for which the unmet medical need is high and there are no effective treatments. Ultragenyx intends to build a sustainable pipeline of safe and effective therapies to address these underserved diseases.  Ultragenyx' lead program, UX001, is being evaluated as a potential treatment for hereditary inclusion body myopathy (HIBM), also known as GNE myopathy. 

The company is led by an experienced management team in rare disease therapeutics.  Ultragenyx is striving toward an improved model for successful rare disease drug development which has the potential to increase efficiency while maintaining appropriate sa
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SOURCE Ultragenyx Pharmaceutical Inc.
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4. Ultragenyx Granted Orphan Drug Designation for UX001 SA-ER for the Treatment of Hereditary Inclusion Body Myopathy (HIBM)
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